Assisted Clinical Application of Gene Therapy for Hemophilia B of Vitalgen Accepted by NMPA
Updated: Feb 15
On April 21, 2022, according to the Chinese CDE (Center for Drug Evaluation) official website, the clinical trial application of VGB-R04 Injection independently developed by BriGene’s partner Shanghai Vitalgen BioPharma Co., Ltd. has been approved by the CDE of National Medical Products Administration (approved project number: CXSL2200055). VGB-R04 Injection treats hemophilia B, which is caused by congenital factor IX deficiency.
VGB-R04 is the first AAV gene therapy product independently developed by Shanghai Vitalgen BioPharma Co., Ltd. Following intravenous injection of VGB-R04, the VGB-R04 expression cassette is delivered to cell nuclei via AAV capsid protein-mediated transfection of liver cells. The VGB-R04 expression cassette exists in the form of cell-free DNA and expresses the highly active natural variant of factor IX (hFIX Padua) protein in liver cells. This liver-expressed and secreted hFIX Padua protein acts in place of the missing congenital coagulation factor IX, thereby correcting the coagulation disorder in hemophilia B patients. hFIX Padua is about 8 times more active compared to the ordinary wild-type FIX, allowing it to perform normal coagulation function at a lower expression level, thus reducing the virus dose amount and improving the safety and efficacy of the product.
A Collective Effort
In partnership with Vitalgen, BriGene provided CGT drug process development and production CDMO services to accelerate the commercialization of the project. BriGene provided Vitalgen with regulation compliant, efficient, and high-quality services including strain library construction, GMP plasmid development and production, GMP AAV development and production, methodology and stability research, and more.
Based on a well-established integrated process platform and abundant technical experience, BriGene has successfully completed the preclinical CMC process development and production of the LX101 project. In the past three years, BriGene has established GMP factories in Jinan and Suzhou (China), and established CMC and R&D centers in Canada and the Un
ited States. As of now, BriGene’s fermentation scales include 5L, 10L, 20L, 200L, 500L, and 2000L and are equipped with advanced purification technology, producing AAV virus titer as high as 1E14VG/L.
BriGene has always been committed to technological innovation. In addition to the production of GMP AAV virus platforms, it has also developed related technology platforms including strain bank construction, cell bank construction, GMP plasmid, GMP lentivirus and CAR-T preparation, as well as quality testing platforms covering biochemistry, microorganisms, and chemistry, with each platform in complete physical isolation to avoid cross-contamination. BriGene’s one-stop platform can fully meet